Rare Daily Staff

Akcea Therapeutics and Ionis Pharmaceuticals said that they received notification from the U.S. Food and Drug Administration that it would not approve the companies’ experimental therapy Waylivra as a treatment for familial chylomicronemia syndrome (FCS), an ultra-rare, genetic, and potentially fatal disease.

FCS is a condition in which patients cannot metabolize triglyceride-rich lipid particles called chylomicrons due to a deficiency in the enzyme lipoprotein lipase. As a result, people with FCS have high levels of triglycerides in the blood, which leads to a range of symptoms including permanent organ damage and potentially fatal attacks of acute pancreatitis.

The companies did not disclose why the FDA chose to not approve the drug. There was expectation among investors that the agency would act in the company’s favor after an advisory committee voted 12 to 8 to recommend its approval despite some concerns about safety among some of the committee members.

“We are extremely disappointed with the FDA’s decision. FCS is an ultra-rare and debilitating disease. Our disappointment extends to the patient and physician community who currently do not have a treatment available to them,” said Paula Soteropoulos, CEO of Akcea Therapeutics. “We continue to feel strongly that Waylivra demonstrates a favorable benefit/risk profile in people with FCS as was reflected in the positive outcome from our Advisory Committee hearing in May. We will continue to work with the FDA to confirm the path forward.”

Results from the phase 3 APPROACH trial, the largest study ever conducted in patients with FCS, show that in comparison to placebo, treatment with Waylivra reduced triglycerides by 77 percent (-94% when compared to placebo). The Endocrine Society and current clinical practice guidelines recommend triglyceride reduction as the goal of treatment for FCS. The most common adverse events in the APPROACH study were injection site reactions and reductions in platelet levels.

Waylivra is also under regulatory review in the European Union and Canada for the treatment of people with FCS. It is also currently in phase 3 clinical development for the treatment of patients with familial partial lipodystrophy, or FPL. Akcea anticipates reporting top-line data from this study in 2019.

August 28, 2018
Photo: Paula Soteropoulos, CEO of Akcea Therapeutics